September 2024

As an early investor in Capricor Therapeutics, CureDuchenne is happy to share that Capricor has announced their intent to apply to the FDA for full approval of Deramiocel (CAP-1002) for the treatment of Duchenne cardiomyopathy.   This BLA (Biologics License Application) filing will be based on existing cardiac data from the Phase 2 HOPE-2 and HOPE-2…

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Capricor Therapeutics announced today its intent to file a Biologics License Application (BLA) seeking full approval of deramiocel for the treatment of Duchenne-cardiomyopathy based on existing cardiac data from their Phase 2 trials of deramiocel (CAP-1002) compared to natural history data. Deramiocel is the company’s novel cell therapy for the treatment of patients diagnosed with…

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I believe that all of us who live with a chronic illness deal with a future full of unknowns. We all wrestle with uncertainty regarding quality of life from year to year, month to month, and even day to day. My chronic illness is limb-girdle muscular dystrophy. The longer we live, the more experiences we…

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SMT-M01, a Duchenne muscular dystrophy (DMD) treatment, was granted orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration (FDA). The cell replacement therapy from Somite Therapeutics uses artificial intelligence (AI) and large, complex data sets to improve production of multiple human cell types. The company plans to begin clinical trials…

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I’m going gray. Wait, I should clarify that I’ve been coloring my hair since I was 25. It started coming in gray long before I became a mom to three sons with Duchenne muscular dystrophy (DMD): Max, 18, Rowen, 15, and Charlie, 13. But after hiding my natural color for 20 years, I’ve decided to…

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We are proud to highlight the National Academies of Sciences, Engineering, and Medicine (NASEM) Consensus Study Report: Regulatory Processes for Rare Disease Drugs in the United States and European Union – Flexibilities and Collaborative Opportunities, with PPMD’s President and CEO Pat Furlong serving on the committee that helped shape its findings. This report outlines crucial…

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Setting Up A Successful School Year PPMD is here to make sure that families within the Duchenne and Becker community have the necessary tools to advocate for their child not only in medical settings, but at school, as well. Together with you and your school’s staff, we want to ensure your child has a successful…

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On September 18, 2024, PPMD hosted an informative community webinar featuring representatives from ITF Therapeutics, PPMD, and the Little Hercules Foundation. The session focused on DUVYZAT (givinostat), a recently approved HDAC inhibitor for the treatment of Duchenne, and the support programs available for patients and families. ITF Therapeutics provided an overview of DUVYZAT, explaining its…

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I’ve had a walker with wheels for a decade. I got it after back surgery in 2014 and used it for a month or so. As a blind person with facioscapulohumeral muscular dystrophy (FSHD), I found it much more difficult to maintain my orientation without direct touch. I was also running the walker into walls,…

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PPMD is thrilled to announce that we have collaborated with a number of patient organizations around the globe to provide Leiden University Medical Center (LUMC), together with the Duchenne Data Foundation (DDF), funding to further continue deep phenotyping of the brain in Duchenne and Becker muscular dystrophy. This research project, set to commence on October…

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